CA3179051A1 - Compositions d'oligonucleotides a double brin et methodes associees - Google Patents

Compositions d'oligonucleotides a double brin et methodes associees

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Publication number
CA3179051A1
CA3179051A1 CA3179051A CA3179051A CA3179051A1 CA 3179051 A1 CA3179051 A1 CA 3179051A1 CA 3179051 A CA3179051 A CA 3179051A CA 3179051 A CA3179051 A CA 3179051A CA 3179051 A1 CA3179051 A1 CA 3179051A1
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Canada
Prior art keywords
double stranded
present disclosure
methods relating
stranded oligonucleotide
oligonucleotide compositions
Prior art date
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Pending
Application number
CA3179051A
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English (en)
Inventor
Chandra Vargeese
Naoki Iwamoto
Luciano H. APPONI
David Charles Donnell Butler
Pachamuthu Kandasamy
Subramanian Marappan
Snehlata Tripathi
Wei Liu
Mugdha BEDEKAR
Vinod VATHIPADIEKAL
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Wave Life Sciences Pte Ltd
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Wave Life Sciences Pte Ltd
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Publication date
Application filed by Wave Life Sciences Pte Ltd filed Critical Wave Life Sciences Pte Ltd
Publication of CA3179051A1 publication Critical patent/CA3179051A1/fr
Pending legal-status Critical Current

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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/314—Phosphoramidates
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
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    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3233—Morpholino-type ring
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
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    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/345—Spatial arrangement of the modifications having at least two different backbone modifications
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
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  • Biochemistry (AREA)
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  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)

Abstract

La présente invention concerne des oligonucléotides à double brin, des compositions et des méthodes associées. La présente invention porte sur la reconnaissance du fait que des éléments structuraux d'oligonucléotides à double brin, tels que la séquence de base, des modifications chimiques (par exemple, des modifications de sucre, de base et/ou de liaisons internucléotidiques) ou des motifs associés, et/ou une stéréochimie (par exemple, la stéréochimie des centres chiraux du squelette (liaisons internucléotidiques chirales)), et/ou des motifs associés, peuvent avoir un impact significatif sur les propriétés et activités d'oligonucléotides, par exemple, l'activité, la stabilité, la distribution, etc., d'interférence ARN (ARNi). La présente invention concerne également des méthodes de traitement de maladies à l'aide des compositions d'oligonucléotides à double brin fournies, par exemple, en interférence ARN.
CA3179051A 2020-05-22 2021-05-24 Compositions d'oligonucleotides a double brin et methodes associees Pending CA3179051A1 (fr)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063029060P 2020-05-22 2020-05-22
US63/029,060 2020-05-22
PCT/IB2021/000351 WO2021234459A2 (fr) 2020-05-22 2021-05-24 Compositions d'oligonucléotides à double brin et méthodes associées

Publications (1)

Publication Number Publication Date
CA3179051A1 true CA3179051A1 (fr) 2021-11-25

Family

ID=76845263

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3179051A Pending CA3179051A1 (fr) 2020-05-22 2021-05-24 Compositions d'oligonucleotides a double brin et methodes associees

Country Status (11)

Country Link
US (1) US20230203484A1 (fr)
EP (1) EP4153747A2 (fr)
JP (1) JP2023526533A (fr)
KR (1) KR20230016201A (fr)
CN (1) CN115885042A (fr)
AU (1) AU2021274944A1 (fr)
BR (1) BR112022023465A2 (fr)
CA (1) CA3179051A1 (fr)
IL (1) IL298406A (fr)
MX (1) MX2022014606A (fr)
WO (1) WO2021234459A2 (fr)

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JP7296882B2 (ja) 2016-11-23 2023-06-23 ウェイブ ライフ サイエンシズ リミテッド ホスホラミダイト及びオリゴヌクレオチド合成のための組成物及び方法
AU2021213822A1 (en) 2020-01-31 2022-09-22 Avilar Therapeutics, Inc. ASGPR-binding compounds for the degradation of extracellular proteins
WO2023152371A1 (fr) 2022-02-14 2023-08-17 Proqr Therapeutics Ii B.V. Oligonucléotides guides pour l'édition d'acides nucléiques dans le traitement de l'hypercholestérolémie
WO2024013360A1 (fr) 2022-07-15 2024-01-18 Proqr Therapeutics Ii B.V. Oligonucléotides chimiquement modifiés pour édition d'arn médiée par adar
WO2024013361A1 (fr) 2022-07-15 2024-01-18 Proqr Therapeutics Ii B.V. Oligonucléotides pour édition d'arn médiée par adar et leur utilisation
GB202215614D0 (en) 2022-10-21 2022-12-07 Proqr Therapeutics Ii Bv Heteroduplex rna editing oligonucleotide complexes
CN117534717A (zh) * 2024-01-09 2024-02-09 凯莱英生命科学技术(天津)有限公司 5′-(e)-乙烯基磷酸酯的合成方法

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